Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6
Open accessAbstract
Retrograde transport of viral vectors in the rodent spinal cord provides a powerful means to administer a therapeutic transgene from the innervated musculature. With the aim of scaling up this approach to non-human primates, we have injected recombinant adeno-associated vectors (rAAV) serotype 6 expressing enhanced green fluorescent protein (eGFP) into the gastrocnemius muscle of African green monkeys to determine whether this results in efficient transgene delivery to lumbar motor neurons. Cells expressing eGFP were observed across more than 1 cm of the spinal cord 4 weeks after intramuscular injection, reaching more than half of motor neurons in some cross-sections. Furthermore, quantitative PCR on the spinal cord tissue confirmed that eGFP expression within motor neurons was due to bona fide retrograde transport of the vector genome from the muscle. Although infiltrations of macrophages and lymphocytes were observed in the rAAV2/6-injected muscle, there was no detectable immune response within the transduced region of the spinal cord. These findings imply that retrograde delivery of rAAV serotype 6 in a primate species constitutes a non-invasive and robust approach to transduce motor neurons, a crucial target cell population in neurodegenerative disorders, such as amyotrophic lateral sclerosis and spinal muscular atrophy.
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After intrathecal delivery, vectors stay predominantly in the lower spine.
“The vectors stay predominantly in the lower spine in intrathecal delivery administrations.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [93]
Use of intrathecal delivery for gene therapy vectors has been limited to studies involving spinal cord and peripheral nerve disease.
“Use of intrathecal delivery for gene therapy vectors has been limited to studies involving spinal cord and peripheral nerve disease.”
From AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease (2018) · cites this paper as [93]
IM delivery of AAV6 showed about 50% transduction of spinal motor neurons in NHPs.
“Moreover, IM delivery of AAV6 showed about 50% transduction of spinal motor neurons in NHPs [105].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [105]
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- Authors
- Towne, C., Schneider, B. L., Kieran, D., Redmond, D. E., & Aebischer, P.
- Affiliations
- École Polytechnique Fédérale de Lausanne (CH)
- Yale University (US)
- Corresponding author
- Aebischer, P
- ORCID
- Schneider, B L
- Venue
- Gene Therapy
- Published
- 2009-09-03
- Volume
- 17
- Issue
- 1
- Pages
- 141-146
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- OpenAlex
- W2151102772
- Cited by
- 124
- References
- 25
- Citations by year
- 2026: 2 · 2025: 2 · 2024: 4 · 2023: 6 · 2022: 4 · 2021: 7 · 2020: 5 · 2019: 11 · 2018: 5 · 2017: 8 · 2016: 11 · 2015: 6 · 2014: 7 · 2013: 14 · 2012: 7
- FWCI
- 6.22
- Type
- journal article
- Language
- en
- Open access
- green
- infoscience.epfl.ch
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Infoscience (Ecole Polytechnique Fédérale de Lausanne) (submittedVersion)
- Infoscience (Ecole Polytechnique Fédérale de Lausanne) (submittedVersion, cc-by-nc-nd)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- motor neurons, intramuscular injection, non-human primates, vervet monkey, spinal cord, retrograde transport, EGFP expression, neurodegenerative diseases, amyotrophic lateral sclerosis, spinal muscular atrophy, immune response
- MeSH terms
- Dependovirus, Animals, Chlorocebus aethiops, Genetic Vectors, Injections, Intramuscular, Motor Neurons, Spinal Cord, Transduction, Genetic, Transgenes, Green Fluorescent Proteins
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